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Bridge Medicines Enters Exclusive License Agreement with The Rockefeller University for Novel Epigenetic Leukemia Program

On March 16, 2020 Bridge Medicines and The Rockefeller University reported an exclusive license agreement to develop novel inhibitors of ENL-YEATS for the treatment of acute leukemias such as Acute Myelogenous Leukemia (AML) and potentially solid tumors (Press release, Bridge Medicines, MAR 16, 2020, View Source [SID1234555625]).

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ENL-YEATS is an epigenetic "reader" protein that emerged from breakthrough science conducted in the laboratory of Lasker Award winner C. David Allis, Ph.D. The work was led by scientist Liling Wan, Ph.D., who was a postdoctoral fellow in the Allis lab at the time. Dr. Allis is the Joy and Jack Fishman Professor, Laboratory of Chromatin Biology and Epigenetics; Dr. Wan was recently appointed Assistant Professor of Cancer Biology and Assistant Investigator of the Abramson Family Cancer Research Institute, Perelman School of Medicine, University of Pennsylvania.

Epigenetic pathways have been shown to regulate cancer cell proliferation and growth, which have recently proven to be successful drug targets. Drs. Wan and Allis found that ENL-YEATS was essential for leukemogenesis and leukemia maintenance by assuring transcription of several oncogenes, including MYC, a regulator gene.

The researchers also discovered a role for ENL-YEATS in Wilms Tumor, a rare kidney cancer found in children, suggesting that ENL-YEATS may be a driver in tumor types beyond acute leukemias.

The Tri-Institutional Therapeutics Discovery Institute, Inc. (Tri-I TDI) advanced the pioneering discoveries from the Allis Laboratory into early-stage drug molecules. Tri-I TDI’s mission is to work with innovative scientists at its founding institutions, The Rockefeller University, Memorial Sloan Kettering Cancer Center, and Weill Cornell Medicine, in a close collaboration with Takeda Pharmaceutical Company Ltd, to accelerate the discovery of new drug molecules.

For this ENL-YEATS program, the scientists at Tri-I TDI, in collaboration with Drs. Wan and Allis, were able to synthesize promising, patented, new molecules that may represent future new drug products. Bridge Medicines will further develop and advance these molecules through preclinical and clinical trials in an effort to bring a new treatment to patients.

"The ENL-YEATS discovery is a major scientific advancement in the area of cancer treatment, and we are thrilled to bring this important program into our portfolio," said Bill Polvino, CEO of Bridge Medicines. "We are encouraged by the promising data developed by these premier academic scientists and look forward to rapidly accelerating the development of this program, in keeping with our mission of translating brilliant discoveries into innovative medicines."

Dr. Allis added, "The ENL-YEATS program offers great promise and hope for patients with AML and Wilms Tumor. We are gratified to have Bridge Medicines partner with us in advancing this program and look forward to seeing it develop into a successful epigenetic therapy to combat these and potentially other diseases."

About AML
Acute Myeloid Leukemia (AML) is a cancer of the myeloid line of blood cells, characterized by the rapid growth of abnormal cells that build up in the bone marrow and blood, interfering with normal blood cells. AML progresses rapidly and is typically fatal within weeks or months if left untreated. Current standard of care is chemotherapy, followed by radiation therapy or stem cell transplant. In 2015, AML affected about one million people and resulted in 147,000 deaths globally, and accounts for about 1.8% of cancer deaths in the United States.

About Wilms Tumor
Wilms Tumor, also known as nephroblastoma, is a cancer of the kidneys that typically occurs in children. It is named after Max Wilms, a German surgeon (1867-1918) who first described it. Approximately 650 cases are diagnosed in the U.S., annually. The majority of cases occur in children with no associated genetic syndromes; however, a minority of children with Wilms Tumor have a congenital abnormality.

Medivir and Tango Therapeutics Sign License Agreement for Preclinical Asset

On March 16, 2020 Medivir AB (Nasdaq Stockholm: MVIR) reported that it has entered into a license agreement with US biotech company Tango Therapeutics for one of Medivir’s preclinical research programs (Press release, Medivir, MAR 16, 2020, View Source [SID1234555624]). Under the terms of the agreement, Medivir will receive an undisclosed upfront payment and is eligible to receive multiple undisclosed development and commercial milestones as well as low single-digit royalties on future products.

"We are pleased to see one of our promising preclinical assets finding a new home for further exploration by a very exciting company" said Dr Christina Herder, EVP & COO of Medivir.

"Synthetic lethality as the basis for a cancer drug target discovery engine holds tremendous promise, exemplified by the number of novel context-dependent cancer drug targets we have discovered using this approach in the past few years," said Barbara Weber, MD, Tango’s President and Chief Executive Officer. "In-licensing this program from Medivir will help us to capitalize on a novel synthetic lethal interaction and accelerate an important program."

AcelRx Pharmaceuticals Reports Fourth Quarter and Full Year 2019 Financial Results

On March 16, 2020 AcelRx Pharmaceuticals, Inc. (Nasdaq: ACRX), (AcelRx), a specialty pharmaceutical company focused on the development and commercialization of innovative therapies for use in medically supervised settings, reported its fourth quarter and full year 2019 financial results (Press release, AcelRx Pharmaceuticals, MAR 16, 2020, View Source [SID1234555623]).

"After only two quarters in 2019 with an expanded sales team, exceeding last year’s formulary and REMS objectives is a solid indicator of the growing acceptance of DSUVIA as a treatment option for the management of acute pain. We fully expect DSUVIA’s success to continue and look forward to the Department of Defense’s Milestone C meeting, which we believe will provide even more opportunities," continued Angotti. "I’m also excited about the announcement earlier today related to our acquisition of Tetraphase Pharmaceuticals, Inc., which will add a key, high growth product to the portfolio, enhancing our offering to healthcare institutions, while increasing the productivity of the commercial teams."

Fourth Quarter and Recent Highlights

Announced an agreement to acquire Tetraphase Pharmaceuticals in a stock for stock transaction at an exchange ratio of 0.6303 shares of AcelRx for each share of Tetraphase, valuing Tetraphase at $14.4 million as of the close of trading on March 13, 2020. Also entered into a co-promotion agreement to immediately begin realizing commercial combination benefits prior to closing of the acquisition.
The Company is on track to achieve its previously communicated target of 465 REMS-certified facilities and formulary approvals by the end of 2020. As of March 15, 2020, 218 healthcare facilities are now REMS-certified and able to purchase DSUVIA and 223 formulary approvals have been achieved.
Confirmed timing for April 2020 DSUVIA Milestone C meeting with the Department of Defense, with procurement recommendation expected post-meeting.
Announced an agreement with Brigham and Women’s Hospital for an investigator-initiated study led by Richard D. Urman MD, MBA, Associate Professor of Anesthesia and co-director of the Center for Perioperative Research at Brigham and Women’s Hospital and Harvard Medical School. The study plans to examine the perioperative use of DSUVIA in the analgesic regimen for spine surgery.
Financial Information

Cash, cash equivalents and short-term investments balance of $66.1 million as of December 31, 2019;
Fourth quarter 2019 net revenues were $0.5 million, and for the full year 2019 were $2.3 million, as previously announced;
Combined R&D and SG&A expenses for the fourth quarter of 2019 totaled $13.8 million compared to $10.4 million for the fourth quarter of 2018. Excluding stock-based compensation expense, these amounts were $12.6 million for the fourth quarter of 2019 compared to $9.2 million for the fourth quarter of 2018. R&D and SG&A expenses for the year ended December 31, 2019 totaled $49.7 million compared to $33.9 million for the year ended December 31, 2018. Excluding stock-based compensation expense, these figures were $44.9 million for the year ended December 31, 2019 compared to $29.1 million for the year ended December 31, 2018. The increase in combined R&D and SG&A expenses is primarily due to increased personnel-related expenses for the commercial launch of DSUVIA. See the "Reconciliation of Non-GAAP Financial Measures" table below for a reconciliation of the non-GAAP operating expenses described above to their related GAAP measures;
Net cash outflow for the fourth quarter of 2019 was $14.3 million, including $0.6 million in debt service, and for the year-ended December 31, 2019 was $39.6 million, and;
For the fourth quarter of 2019, net loss was $14.4 million, or $0.18 per basic and diluted share, compared to $12.6 million, or $0.18 per basic and diluted share, for the fourth quarter of 2018. Net loss for the year ended December 31, 2019 was $53.2 million, or $0.67 per basic and diluted share, compared to $47.1 million, or $0.81 per basic and diluted share, for the year ended December 31, 2018.
2020 Guidance
As previously announced, the Company’s year-end goals include obtaining 465 REMS-certified facilities and 465 formulary approvals in 2020. Quarterly combined R&D and SG&A expense in 2020 is expected to range from $10 million to $13 million, depending on the quarter, and includes approximately $1 million of non-cash stock-based compensation per quarter ($9 million to $12 million excluding stock-based compensation expense). Annual debt service is expected to approximate $6 million. Annual capital expenditures are expected to range from $4-$5 million attributed mainly to the installation of a new high-volume, automated packaging line at our contract manufacturer. These amounts do not consider the impact from the previously announced acquisition of Tetraphase Pharmaceuticals but reflect the benefits of the co-promotion agreement.

2020 financial guidance is based on the Company’s current expectations and are forward-looking statements. Actual results could differ materially depending on market conditions and the factors set forth under the "Forward-Looking Statements" heading below.

Webcast and Conference Call Information
As previously announced, AcelRx will host a live webcast Monday, March 16, 2020 at 8:30 a.m. Eastern Time (5:30 a.m. Pacific Time) to discuss these financial results and provide other corporate updates. The webcast is accessible by visiting the Investors page of the Company’s website at www.acelrx.com and clicking on the webcast link. The webcast will be accompanied by a slide presentation. Investors who wish to participate in the conference call may do so by dialing (866) 361-2335 for domestic callers, (855) 669-9657 for Canadian callers or (412) 902-4204 for international callers. A webcast replay will be available on the AcelRx website for 90 days following the call by visiting the Investor page of the Company’s website at www.acelrx.com.

About DSUVIA (sufentanil sublingual tablet), 30 mcg
DSUVIA, known as DZUVEO in Europe, approved by the FDA in November 2018, is indicated for use in adults in certified medically supervised healthcare settings, such as hospitals, surgical centers, and emergency departments, for the management of acute pain severe enough to require an opioid analgesic, and for which alternative treatments are inadequate. DSUVIA was designed to provide rapid analgesia via a non-invasive route and to eliminate dosing errors associated with intravenous (IV) administration. DSUVIA is a single-strength solid dosage form administered sublingually via a single-dose applicator (SDA) by healthcare professionals. Sufentanil is an opioid analgesic previously only marketed for IV and epidural anesthesia and analgesia. The sufentanil pharmacokinetic profile when delivered sublingually avoids the high peak plasma levels and short duration of action observed with IV administration. The European Commission approved DZUVEO for marketing in Europe in June 2018 and the Company is currently in discussions with potential European marketing partners.

EnGeneIC Announces Publication in Cancer Cell of a Scientific Paper Highlighting the Ability of EDV™ Nanocells to Mount Dual Assault on Cancer Cells

On March 16, 2020 EnGeneIC Limited, a clinical-stage biopharmaceutical company advancing its proprietary EDV nanocell platform for targeted cyto-immunotherapy in cancer, reported that the prestigious peer-reviewed journal Cancer Cell has published a scientific paper, authored by EnGeneIC’s research team, entitled "Cyto-Immuno-Therapy for Cancer: A Pathway Elicited by Tumor-Targeted, Cytotoxic Drug-Packaged Bacterially Derived Nanocell (Press release, EnGeneIC, MAR 16, 2020, View Source [SID1234555622])." The paper describes how the EnGeneIC Dream Vector (EDV) technology stimulates both an innate and an adaptive immune response and creates a dual method of action that could result in long-term survival. Additionally, Cancer Cell will display the EDV’s immunotherapy mechanism of action as this month’s cover art.

The immuno-oncology field has been focused largely on chimeric antigen receptor T-cell (CAR-T) therapies or checkpoint inhibitors, both designed to enable cytotoxic T-cells to target cancer cells. However, research shows that T-cells are only part of the story. Achieving a robust anti-tumor immune response requires the stimulation of several different types of immune cells. The EnGeneIC research paper describes how the company’s targeted EDV nanocells combine cytotoxicity by virtue of their chemotherapy payload, and at the same time incite a totally novel and more complete anti-tumor immune response. The EDVs stimulate an innate immune response by activating macrophages, natural killer cells and dendritic cells with a subsequent adaptive immune response resulting from recruitment of specific CD8+ tumor killing T-cells into the tumor microenvironment. This work paves the way for treatment of even drug-resistant, end-stage cancers across many tumor types with little or no toxicity, and for a fraction of the cost compared to other immunotherapies capable of treating only a limited number of cancer indications.

Himanshu Brahmbhatt, Ph.D., co-Chief Executive of EnGeneIC and the study’s senior author, stated, "The EnGeneIC team is excited by the publication of its latest scientific paper in Cancer Cell. For the first time, we show that one therapeutic has the ability to carry a toxic payload to kill cancer cells and also jump-start the depleted immune system. This two-prong attack is showing preliminary success in early human trials."

Clinical trials are now underway in Australia evaluating EDV in multiple cancer indications, including pancreatic cancer patients with stage IV disease who have exhausted curative treatment options. The trial also has a second all-comers cohort for patients suffering with a variety of other a late-stage EGFR-expressing solid tumors.

Dr. Brahmbhatt continued, "EDV treatment is not a piecemeal approach to cancer – all the key immune cells required for an assault on the tumor are stimulated. When we depleted each of these cell types, the anti-tumor effect was diminished. As well as delineating the novel mechanism of action in mouse models of cancer, our paper describes two patients who responded with an anti-tumor immune response. We look forward to advancing the development of our EDV nanocell technology platform to bring hope to those patients most in need."

The Cancer Cell article is available to view at the following link: View Source

Immunic, Inc. Reports Year End 2019 Financial Results and Highlights Recent Achievements

On March 16, 2020 Immunic, Inc. (Nasdaq: IMUX), a clinical-stage biopharmaceutical company focused on developing best-in-class, oral therapies for the treatment of chronic inflammatory and autoimmune diseases, reported financial results for the year ended December 31, 2019 and highlighted recent achievements (Press release, Immunic, MAR 16, 2020, View Source [SID1234555621]).

"In addition to the closing of our stock-for-stock exchange transaction with Vital Therapies, Inc., which provided our Nasdaq listing and a significant capital infusion, 2019 was punctuated by the attainment of other important milestones," stated Daniel Vitt, Ph.D., Chief Executive Officer and President of Immunic. "Most notably, we completed enrollment for our phase 2 EMPhASIS trial of our lead compound, IMU-838, for patients with relapsing-remitting multiple sclerosis (RRMS), roughly nine months ahead of our initial plan. With preclinical data showing a solidly superior profile compared to DHODH inhibitor, teriflunomide, IMU-838 holds promise as an important new, best-in-class oral therapy for RRMS and other underserved immunologic diseases. We anticipate reporting top-line results in the third quarter of this year and believe that positive data could allow us to move quickly into a pivotal phase 3 trial.

"We also reported progress for IMU-838 in moderate-to-severe ulcerative colitis (UC) and primary sclerosing cholangitis (PSC), with a positive phase 2 interim dosing analysis in UC and the initiation of our investigator-sponsored, phase 2 trial in PSC, led by the Mayo Clinic. Given that the interim dosing analysis from the CALDOSE-1 study in patients with UC showed that it was likely not ineffective at the lowest 10 mg dose and that none of the three dose levels revealed unacceptable intolerance, the trial was expanded to 240 patients and top-line data is expected in the fourth quarter of 2021."

Dr. Vitt continued, "Our earlier stage programs have also advanced, with the dosing of healthy volunteers in several dose cohorts in our phase 1 trial of IMU-935, a potentially best-in-class inverse agonist of RORγt. Additionally, we plan to complete the preclinical and manufacturing activities required in order to initiate phase 1 clinical studies of IMU-856, during the first half of this year, having just announced in January 2020 the exercise of our option for the exclusive worldwide license to IMU-856 from Daiichi Sankyo, Co., Ltd. We are highly encouraged by the data we have generated thus far for our programs, most especially for IMU-838, and look forward to additional success in 2020."

Recent Highlights

January 2020: Exercised option from Daiichi Sankyo Co., Ltd. for the exclusive worldwide license to a group of compounds, designated by Immunic as IMU-856, aimed at restoring intestinal barrier function.
December 2019: Presented data, for the first time, on IMU-856, at the Crohn’s and Colitis Foundation IBD Innovate Conference. The presentation highlighted preclinical data indicating that the compound potentially restores intestinal barrier function without impairing the immune system. This represents a new and possibly disruptive approach for the treatment of intestinal diseases.
November 2019: Expanded the Board of Directors to seven members with the appointment of biotechnology executive Barclay "Buck" A. Phillips.
October 2019: Expanded the Board of Directors to six members with the appointment of industry veteran Tamar Howson.
October 2019: Announced early completion of patient enrollment (nine months ahead of initial schedule) for the phase 2 EMPhASIS trial of IMU-838, for the treatment of RRMS.
Upcoming Anticipated Clinical Milestones

Top-line data from the phase 2 EMPhASIS trial in RRMS is expected to be available in the third quarter of 2020.
Completion of the preclinical and manufacturing activities that are necessary for the initiation of phase 1 clinical studies of IMU-856 is expected during the first half of 2020.
The current, single ascending dose trial of IMU-935 is planned to be followed by a phase 1 multiple ascending dose trial in healthy volunteers and a phase 1 trial in patients with mild-to-moderate psoriasis; both are expected to start during the first half of this year.
Top-line data from the phase 2 CALDOSE-1 trial in UC is expected to be available during the fourth quarter of 2021.
Financial and Operating Results

Research and Development (R&D) Expenses were $22.5 million for the year ended December 31, 2019, compared to $9.6 million for the same period during 2018, an increase of $12.9 million. The increase is primarily due to (i) higher external development costs for the company’s IMU-838 program for the phase 2 clinical trial in patients with RRMS and UC and preparation costs related to the phase 2 clinical trial for patients with Crohn’s disease totaling $8.3 million, (ii) an increase of drug supply costs to support clinical trials of IMU-838 totaling $1.5 million, (iii) a contingent payment under the asset purchase agreement with 4SC AG settled in stock valued at $1.5 million, (iv) external R&D costs related to the company’s IMU-856 program of $1.1 million and (v) $0.5 million related to increases across numerous categories.
General and Administrative (G&A) Expenses were $14.5 million for the year ended December 31, 2019, compared to $2.4 million for the same period during 2018, an increase of $12.1 million. The increase is primarily due to (i) one-time costs related to the stock-for-stock exchange transaction completed on April 12, 2019 with Vital Therapies, including $6.4 million of stock-based compensation for executives, key employees and members of the board and $1.7 million of investment banking and legal fees, (ii) $2.6 million related to becoming a public company, including directors and officers liability insurance, audit and legal fees and personnel costs for executives and staff in the U.S. corporate headquarters, (iii) $0.5 million related to travel and (iv) $0.9 million related to increases across numerous categories.
Other Income for the year ended December 31, 2019 was $2.1 million compared to $450,000 for the same period of 2018, an increase of $1.6 million. The increase is primarily attributable to a $0.9 million year-over-year increase in reimbursement of research and development expenses in connection with the option and license agreement with Daiichi Sankyo Co., Ltd., the $0.3 million as a result of the sale of certain of Vital Therapies’ clinical development-related assets and related intellectual property, $0.3 million related to research and development tax incentives for clinical trials in Australia and $0.1 million related to interest income.
Net Loss for year ended December 31, 2019 was approximately $34.9 million, or $4.52 per basic and diluted share, based on 7,722,269 weighted average common shares outstanding, compared to a net loss of approximately $11.5 million, or $13.63 per basic and diluted share, based on 846,953 weighted average common shares outstanding for the year ended December 31, 2018. Substantially all of the company’s operating losses resulted from expenses incurred in connection with its R&D programs and from G&A costs associated with operations.
Cash and Cash Equivalents, as of December 31, 2019, of $29.4 million, is expected to fund the company’s operations into the first quarter of 2021.